Lenti-D Hematopoietic Stem Cell Gene Therapy Stabilizes Neurologic Function in Boys with Cerebral Adrenoleukodystrophy
نویسندگان
چکیده
منابع مشابه
Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy.
X-linked adrenoleukodystrophy (ALD) is a severe brain demyelinating disease in boys that is caused by a deficiency in ALD protein, an adenosine triphosphate-binding cassette transporter encoded by the ABCD1 gene. ALD progression can be halted by allogeneic hematopoietic cell transplantation (HCT). We initiated a gene therapy trial in two ALD patients for whom there were no matched donors. Autol...
متن کاملHematopoietic stem cell gene therapy.
Gene therapy applications that target hematopoietic stem cells (HSCs) offer great potential for the treatment of hematologic disease. Despite this promise, clinical success has been limited by poor rates of gene transfer, poor engraftment of modified cells, and poor levels of gene expression. We describe here the basic approach used for HSC gene therapy, briefly review some of the seminal clini...
متن کاملNeurocognitive Trajectory of Boys Who Received a Hematopoietic Stem Cell Transplant at an Early Stage of Childhood Cerebral Adrenoleukodystrophy.
Importance Untreated childhood cerebral adrenoleukodystrophy (cALD) is a fatal disease associated with progressive cerebral demyelination and rapid, devastating neurologic decline. The standard of care to enhance long-term survival and stabilize cerebral disease is a hematopoietic stem cell transplant (HSCT). Neurologic outcomes are better when HSCT occurs at an earlier stage of cALD, yet there...
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Human gene therapy trials have begun, and by early reports are a success [1]. The initial clinical trial which demonstrated the safety of infusing genetically marked lymphocytes into humans was reported in 1990 [2]. This paved the way for researchers at the National Institutes of Health (NIH) :o begin the first therapeutic trials of treating adenosine deaminase deficient patients with genetical...
متن کاملPediatric Hematopoietic Stem Cell Transplantation
The introduction and evolution of hematopoietic stem cell transplantation (HSCT) could be traced back to 1950s, to the studies on interactions among irradiation, covering spleen and bone marrow from it and injection of bone marrow cells. Today, HSCT is considered a well-established, effective and promising means of therapy for various malignant and non-malignant medical conditions, both in chil...
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ژورنال
عنوان ژورنال: Biology of Blood and Marrow Transplantation
سال: 2020
ISSN: 1083-8791
DOI: 10.1016/j.bbmt.2019.12.103